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Taysha Gene Therapies Reports Positive Clinical Efficacy and Safety Data for High Dose Cohort and Long-term Durability Data for TSHA-120 in Giant Axonal Neuropathy
Efficacy data for high dose cohort demonstrated clinically meaningful and statistically significant improvement in MFM32 by Year 1 compared to natural
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nEfficacy data for high dose cohort demonstrated clinically meaningful and statistically significant improvement in MFM32 by Year 1 compared to natural history (n=3)\n\nLong-term durability data across all therapeutic dose cohorts demonstrated a 10-point improvement in mean change in MFM32 by Year 3 compared to estimated natural history decline of 24 points (n=5)\n\nBiopsy data in five of six patient samples analyzed to date confirmed active regeneration of nerve fibers following treatment with TSHA-120 (n=6)\n\nTSHA-120 was safe and well-tolerated supported by 53 patient-years of clinical data\n\nConference call and live webcast today at 8:00 AM Eastern Time\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported positive clinical efficacy and safety data for the high dose cohort of 3.5x1014 total vg, as well as long-term durability data across all therapeutic doses of TSHA-120 in giant axonal neuropathy (GAN).\n\n“The totality of data generated by TSHA-120 to date support our plans to engage with major regulatory agencies in order to discuss pathways for registration and we look forward to providing a regulatory update later this year,” noted RA Session II, President, Founder and CEO of Taysha. “In the interim, we are finalizing our commercial strategy with a focus on patient identification, disease awareness and payor engagement for the estimated 5,000 affected patients in addressable markets.”\n\nKey Clinical Data Support Durable, Clinically Meaningful and Statistically Significant Slowing of Disease Progression Across All Therapeutic Cohorts (1.2x1014 total vg, 1.8x1014 total vg and 3.5x1014 total vg)\n\n\nHigh Dose Cohort Data (3.5x1014 total vg) by Year 1: 5-point improvement in the change in rate of decline in MFM32 score for the high dose cohort compared to natural history decline of 8 points by Year 1 (n=3, p = 0.04)\n\n\nAnalysis of All Therapeutic Dose Cohorts by Year 1: 7-point improvement in the change in rate of decline in MFM32 score across all therapeutic dose cohorts compared to natural history decline of 8 points by Year 1 (n=12, p","lengt...